FDA Advisory Committee Deems Potential Barth Syndrome Drug “Effective”
Earlier this month, an FDA Advisory Committee voted 10-6 supporting approval of Stealth BioTherapeutics’ elamipretide for treating Barth syndrome, a rare mitochondrial disease that occurs mostly in males affecting the heart, immune system, and muscles, amongst other things.
“The committee’s decision reinforces our belief that, when it comes to families experiencing mitochondrial disease, patients deserve access to any therapy proven to safely improve quality of life for these devastating diseases,” said UMDF’s Interim Managing Director and Science and Alliance Officer Dr. Philip Yeske, who was on-hand for the historic vote.
He also praised the work of the Barth Syndrome Foundation and the many families and clinicians who testified.
“The open public hearing portion of the meeting was extremely powerful and seemingly played a critical role in turning the tide toward a recommendation for approval. I think that’s a reflection of the closeness of this community, and the hard work of the Barth Syndrome Foundation.”
The advisory committee’s recommendation helps guide the FDA’s decision, with a final determination on elamipretide expected by January 29, 2025.
Related Posts:
UMDF Reacts to FDA Advisory Committee’s Elamipretide Recommendation | UMDF.orgUMDF’s Comment to FDA’s Advisory Committee Regarding Elamipretide | UMDF.org
In Other News in DC
Members of Congress are on the campaign trail and are set to return for the lame duck session on November 12, 2024. Upon their return, the primary focus will be addressing government funding, which is currently operating under a continuing resolution (CR) that expires on December 20. Appropriators are in the process of negotiating the FY 2025 spending package, but the outcome of the election may influence whether another CR, extending into 2025, will be needed or if the process can be finalized before the 119th Congress is sworn in.
The possibility of a healthcare package remains uncertain, with its scope depending on the post-election balance of power between Congress and the administration. We will be closely monitoring efforts to include key healthcare priorities that UMDF has advocated for, many of which have seen success in the committee process and floor votes. Specifically, the House of Representatives passed legislation to reauthorize the Pediatric Disease Priority Review Voucher (PRV) program for five years. This program incentivizes the development of therapies for rare pediatric diseases, leading to more than 50 new treatment options in its first 12 years. Additionally, the House passed the bipartisan Accelerating Kids’ Access to Care Act (H.R. 4758), aimed at easing barriers for Medicaid patients seeking out-of-state care. The Energy and Commerce Committee has also advanced a two-year extension of Medicare telehealth flexibilities, setting the stage for a vote by the full House, following similar progress in the health subcommittee and the House Ways and Means Committee. UMDF continues to urge Congress to include these priorities in any year-end package.
As always, visit umdf.org/advocacy to see how you can take advocacy action today to support the mitochondrial disease community.